A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults with C9ORF72-Associated Amyotrophic Lateral Sclerosis
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abstract
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The primary objective of this study is to evaluate the safety and
tolerability of BIIB078 in adults with C9ORF72-Amyotrophic Lateral
Sclerosis (ALS). The secondary objectives of this study are to
evaluate the pharmacokinetic profile of BIIB078 and to evaluate the
effects of BIIB078 on clinical function. As the first-in-human
study, the study enrolls a small number of participants in each
cohort. Every participant in a cohort is treated with the same dose
or placebo. The study is designed to evaluate and confirm the safety
of each dose before enrolling and exposing new participants to a
higher dose in the next cohort.
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type of project
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clinical studies
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status
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completed
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start of project
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2018
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end of project
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2021
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study design
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Phase 1 Study, placebo controlled
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responsible person
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Weber, Markus
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